Clinical Trial Shows Efficacy of Rare Disease Treatment

Data recently published in the Orphanet Journal of Rare Diseases showed that long-term improvements and stability can be achieved with taliglucerase alfa in patients with Gaucher disease.

Taliglucerase alfa, commercially known as Eleyso, is developed by Protalix and Pfizer. It was approved by the U.S. Food and Drug Administration (FDA) in May 2012 for use in adults with Gaucher disease, and a supplemental new drug application (sNDA) for pediatric use was granted approval in September of that same year.

Gaucher disease is a genetic lysosomal storage disorder in which a deficiency of the glucocerebrosidase enzyme leads to a buildup of the lipid glucerebrosidase in the lysosomes. The accumulation become encorged Gaucher cells and can cause multisystemic damage in organs and tissues, especially the spleen, liver, bone, platelets, and hemoglobin.

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